Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy. - Test2 - elhamkhani - TriplyDB

Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy.

Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy.

http://www.wikidata.org/entity/Q35221624

about

Molecular Mechanisms of Neurodegeneration in Spinal Muscular Atrophy A large animal model of spinal muscular atrophy and correction of phenotype.Defective control of pre-messenger RNA splicing in human disease Repeated low doses of morpholino antisense oligomer: an intermediate mouse model of spinal muscular atrophy to explore the window of therapeutic response Selective Neuromuscular Denervation in Taiwanese Severe SMA Mouse Can Be Reversed by Morpholino Antisense Oligonucleotides Collaboration for rare disease drug discovery research Established Stem Cell Model of Spinal Muscular Atrophy Is Applicable in the Evaluation of the Efficacy of Thyrotropin-Releasing Hormone Analog.Protective effects of butyrate-based compounds on a mouse model for spinal muscular atrophy.Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy Somatic Therapy of a Mouse SMA Model with a U7 snRNA Gene Correcting SMN2 Splicing jSplice: a high-performance method for accurate prediction of alternative splicing events and its application to large-scale renal cancer transcriptome data.Emerging therapies and challenges in spinal muscular atrophy Single-Cell Analysis of SMN Reveals Its Broader Role in Neuromuscular Disease.GEMINs: potential therapeutic targets for spinal muscular atrophy?Spinal Muscular Atrophy Therapeutics: Where do we Stand?SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice.Big win possible for Ionis/Biogen antisense drug in muscular atrophy.Improvement of SMN2 pre-mRNA processing mediated by exon-specific U1 small nuclear RNA.Spinal muscular atrophy: from tissue specificity to therapeutic strategies Molecular biology. A splicing magic bullet.Neuromuscular disorders: Beefing up the right splice variant to treat spinal muscular atrophy.Identification of small molecule inhibitors of pre-mRNA splicing.Romancing the spliceosome to fight spinal muscular atrophy.mRNA regulation: A patch for a splice.Disease mechanisms and therapeutic approaches in spinal muscular atrophy.Deciphering targeting rules of splicing modulator compounds: case of TG003.Biomarker for Spinal Muscular Atrophy: Expression of SMN in Peripheral Blood of SMA Patients and Healthy Controls Meeting Report: New Directions in the Biology and Disease of Skeletal Muscle 2014 SMN Protein Can Be Reliably Measured in Whole Blood with an Electrochemiluminescence (ECL) Immunoassay: Implications for Clinical Trials The effect of the DcpS inhibitor D156844 on the protective action of follistatin in mice with spinal muscular atrophy.SMN deficiency disrupts gastrointestinal and enteric nervous system function in mice Histopathological Defects in Intestine in Severe Spinal Muscular Atrophy Mice Are Improved by Systemic Antisense Oligonucleotide Treatment.Molecular Basis and Therapeutic Strategies to Rescue Factor IX Variants That Affect Splicing and Protein Function SMN expression is required in motor neurons to rescue electrophysiological deficits in the SMNΔ7 mouse model of SMA.Small Molecules in Development for the Treatment of Spinal Muscular Atrophy.Conserved overlapping gene arrangement, restricted expression, and biochemical activities of DNA polymerase ν (POLN).Low levels of Survival Motor Neuron protein are sufficient for normal muscle function in the SMNΔ7 mouse model of SMA.Normalization of Patient-Identified Plasma Biomarkers in SMNΔ7 Mice following Postnatal SMN Restoration.Rational Design of Small Molecules Targeting Oncogenic Noncoding RNAs from Sequence.Small Molecule-Based Pattern Recognition To Classify RNA Structure.

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Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy.

http://www.wikidata.org/entity/Q35221624

description

2014 nî lūn-bûn

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2014 թուականի Օգոստոսին հրատարակուած գիտական յօդուած

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2014 թվականի օգոստոսին հրատարակված գիտական հոդված

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2014年の論文

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2014年論文

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2014年論文

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2014年論文

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2014年論文

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2014年論文

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2014年论文

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name

Motor neuron disease. SMN2 spl ...... with spinal muscular atrophy.

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Motor neuron disease. SMN2 spl ...... with spinal muscular atrophy.

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type

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Motor neuron disease. SMN2 spl ...... with spinal muscular atrophy.

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Motor neuron disease. SMN2 spl ...... with spinal muscular atrophy.

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prefLabel

Motor neuron disease. SMN2 spl ...... with spinal muscular atrophy.

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Motor neuron disease. SMN2 spl ...... with spinal muscular atrophy.

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SCIENCE.1250127

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Motor neuron disease. SMN2 spl ...... with spinal muscular atrophy.

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Amal Dakka

http://www.w3.org/2001/XMLSchema#string

Anirvan Ghosh

http://www.w3.org/2001/XMLSchema#string

Anthony Turpoff

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Anuradha Bhattacharyya

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Bansri Furia

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Chang-Sun Lee

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Chien-Ping Ko

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Ellen M Welch

http://www.w3.org/2001/XMLSchema#string

Friedrich Metzger

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Gary M Karp

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P2860

Mapping and quantifying mammalian transcriptomes by RNA-Seq

Evidence for the involvement of human DNA polymerase N in the repair of DNA interstrand cross-links

DNA polymerase POLN participates in cross-link repair and homologous recombination

Fast gapped-read alignment with Bowtie 2

The Sequence Alignment/Map format and SAMtools

An in vivo reporter system for measuring increased inclusion of exon 7 in SMN2 mRNA: potential therapy of SMA

The SMN complex, an assemblyosome of ribonucleoproteins

Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of >72,400 specimens

A degron created by SMN2 exon 7 skipping is a principal contributor to spinal muscular atrophy severity

Regulation of SMN protein stability

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10.1126/SCIENCE.1250127

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spinal muscular atrophy